DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)

by | Aug 2, 2023 | 0 comments

DTx Pharma, a biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its Fatty Acid Ligand Conjugated OligoNucleotides (FALCON™) platform, today announced that the FDA has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).

About FDA Orphan Drug Status

Orphan Drug status refers to a special designation granted to pharmaceutical products that are developed to treat rare diseases or conditions. In many countries, including the United States, the European Union, and Japan, orphan drug status provides certain incentives and benefits to pharmaceutical companies to encourage the development of therapies for rare diseases. These incentives may include extended market exclusivity, tax credits, fee waivers, and assistance with the drug approval process. The intention is to promote the development of treatments for diseases that affect a relatively small number of people and may not be financially viable for pharmaceutical companies to pursue without such incentives. To qualify for orphan drug status, a medication must meet specific criteria defined by regulatory authorities, such as having a prevalence below a certain threshold in the population. Once granted orphan drug status, the pharmaceutical company may receive assistance and incentives to facilitate the development and commercialization of the drug, ultimately benefiting patients with rare diseases.

About DTx-1252

DTx-1252 is a potential first-in-class FALCON™ siRNA therapeutic for treatment of CMT1A. By repressing PMP22, DTx-1252 reverses CMT1A in a mouse model that faithfully recapitulates the genetic and clinical manifestations of the disease. DTx-1252 treatment induces remyelination of axons to normal levels, improves relevant electrophysiological measurements and increases muscle mass, grip strength, coordination and agility in preclinical studies.

Important Steps to take!

If you have a confirmed genetic diagnosis of CMT1A, now is the time to make sure you are in HNF’s Global Registry for Inherited Neuropathies and upload your genetic report.


If you do not have a confirmed genetic diagnosis of CMT1A, now is the time to make an appointment with the CMT Genie to pursue a genetic diagnosis.

Make an appointment with the CMT Genie

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